FDA approves allogeneic regulatory T cell-based immunotherapy with HSPC and T cells-vldq for use in matched donor hematopoietic stem cell transplantation for adults with hematologic malignancies
Regulatory approval: On June 30, 2026, the FDA approved Tregzi (HSPC + allogeneic regulatory T cells + conventional T cells; Orca Bio) for use with a myeloablative matched-donor HSCT regimen in adults with hematologic malignancies to support hematopoietic/immunologic reconstitution and improve chronic GVHD-free survival. In the open-label randomized Precision-T trial (NCT05316701) in acute leukemias/MDS, cGVHD-free survival favored Tregzi versus unmanipulated allograft with standard prophylaxis (HR 0.26).
FDA approval (June 30, 2026)
The FDA approved Tregzi (allogeneic regulatory T cell-based immunotherapy with hematopoietic stem and progenitor cells [HSPCs] and T cells-vldq; Orca Bio) for use in matched-donor hematopoietic stem cell transplantation (HSCT) with a myeloablative preparative regimen in adults with hematologic malignancies, for hematopoietic and immunologic reconstitution and to improve chronic GVHD-free survival (cGFS).
Efficacy and safety (Precision-T; NCT05316701)
Approval was supported by Precision-T, a multicenter, open-label, randomized controlled trial in adults with acute leukemias or myelodysplastic syndrome (n=187). Patients received either Tregzi (n=93) followed by tacrolimus alone, or unmanipulated allograft (n=94) with tacrolimus + methotrexate.
Primary endpoint cGFS (time from HSCT to death or moderate-to-severe cGVHD, NIH criteria; blinded independent adjudication): median not estimable with Tregzi vs 7.3 months in control (HR 0.26, 95% CI 0.14–0.47; P<.00001) at median follow-up 8.48 vs 9.03 months.
12-month cumulative incidence of moderate-to-severe cGVHD: 12.6% vs 44.0% (HR 0.19, 95% CI 0.08–0.43; P=.00002).
Common adverse reactions (≥20%) included mucositis, diarrhea, rash, viral infections, and acute GVHD. Label warnings/precautions include graft failure, GVHD, infusion reactions, secondary malignancies/donor-origin malignancies, and transmission of infectious agents.
The application received priority review, orphan drug, and RMAT designations.